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Investigators shape a workable public/private development model

A few weeks ago the FDA joined hands with the National Institutes of Health to announce a new scientific collaboration that top government officials claimed would help accelerate new drug discoveries from the clinic to the bedside.

"We've all been following the remarkable advances in biomedical sciences led by the NIH with great enthusiasm for years," Kathleen Sebelius, Secretary of Health and Human Services, said with great fanfare. "Collaboration between the NIH and the FDA, including support for regulatory science, will go a long way towards fostering access to the safest and most effective therapies for the American people."

But quite a few analysts, myself included, snickered when they caught sight of the collaboration's budget: $6.75 million over three years. That amounts to just a tiny fraction of the average cost of getting a single drug through to an approval. How much could these agencies really accomplish with a shoestring budget?

No one was acting smug last week, though, when the FDA and the NIH joined a unique collaboration with three drug developers--Amgen, Abbott and Pfizer--to launch I-SPY2, a $26 million, five-year study that will attempt to match patients to experimental breast cancer therapies based on their DNA. And more companies will be invited to join in the study.

"I-SPY 2 will provide a path to personalized medicine," Dr. Laura Esserman, a breast cancer specialist who will help leap the collaboration, told Reuters. "We intend that every drug will graduate with a companion marker."

"What's really neat about the I-SPY trial is that Laura Esserman, the PI of the trial, is a breast cancer surgeon here at UCSF and has added so much value to the project because she sees patients early and has a unique opportunity to offer neoadjuvant therapy," Dr. Susan Desmond-Hellmann--the former R&D chief at Genentech and now chancellor at UCSF--told the Pharma Strategy Blog. "Patients are getting their primary therapy before they get surgery, so for imaging and biomarkers--either established or exploratory--it is a fantastic opportunity. The endpoint is pathological complete response, so you can see if the tumor has disappeared or not."

By being able to tell sooner which drugs are working with which patients, investigators say that this new collaboration has the potential to cut years out of a clinical trial for cancer therapies.

Dr. Tufia Haddad, a breast cancer specialist at the University of Minnesota, speculated that I-SPY2 has the potential to cut 10 years out of a 15-year discovery odyssey. "That is a massive difference," she told the Star-Tribune. "And the cost savings, I can't even put a number on it."

For years now the FDA's position on innovation has been simple: Provide plenty of lip service to the concept while bowing to budget realities and doing little to really make a difference. With the I-SPY2 approach, the agency in collaboration with biotech companies can break a brand new path, demonstrating how developers can all gain when they share data openly as they test new technology.

If this project is even half as successful as promised by investigators, this new approach could also forge a new development model that could prove irresistible to others. That's low-cost innovation that benefits everyone.

"I think it's the future," said Dr. Anna Barker, deputy director of the National Cancer Institute. "Government couldn't have done it on their own and these companies couldn't have done it on their own."

This new private/public development model actually has the potential to work. - John Carroll (twitter | email)